OBJECTIVES:

To evaluate the cost-effectiveness of CPX-351(Vyxeos®) vs the standard of care (SoC) for the treatment of adult patients with newly diagnosed (ND) therapy-related Acute Myeloid Leukemia (t-AML) or AML with Myelodysplasia-Related Changes (MRC) in Greece.

METHODS:

A cost-effectiveness model, developed by Jazz Pharmaceuticals, was locally adapted to reflect the natural progression of patients over 60 years old with ND t-AML/AML-MRC, over a lifetime horizon (30 years) from the public payer perspective. Since daunorubicin, used as SoC in the clinical trial was not reimbursed in Greece, idarubicin substituted daunorubicin, adjusted to match equivalent dosing -as suggested by literature- along with cytarabine and considered as SoC in the present analysis. To extrapolate the trial data, parametric models were applied. A decision tree was firstly used to reflect patients’ pathway after entering the model and receiving induction therapy. Afterwards, patients transitioned to a partitioned-survival model which consists of event free, progression and death health states. Cycle length was set to one and eight weeks for the first two and subsequent years, respectively. Clinical data were extracted from relevant clinical trial and published studies. Direct medical costs (€, 2021) including drug acquisition, administration and hematopoietic stem cell transplantation were derived from official national sources. Adverse events and monitoring costs were obtained from literature. Deterministic (DSA) and probabilistic sensitivity analyses (PSA) were conducted.

RESULTS:

CPX-351 was associated with higher QALYs vs SoC (1.64 vs 0.86) at additional costs of €42,021. The generated incremental cost-effectiveness ratio (ICER) was €53,917 per QALY gained, indicating that CPX-351 represents a cost-effective treatment at a willingness-to-pay threshold of €54,000/QALY gained. DSA and PSA confirmed the robustness of the results.

CONCLUSIONS:

CPX-351 provides additional health gains as compared to SoC at a reasonable cost and hence it appears to be a cost-effective treatment option for adults with ND t-AML/AML-MRC in Greece.